CureQ is an NWO-supported consortium (“Nederlandse Wetenschaps Agenda”) aiming a better prediction of and finding possible treatments to delay or prevent the onset of Huntington’s disease (HD) and two forms of spinocerebellar ataxias (SCA-1 and SCA-3)
HD and several forms of SCAs are autosomal dominantly inherited neurodegenerative disorders caused by a CAG repeat expansion in the disease-related gene, leading to the production of mutant proteins. These mutant proteins erroneously have regions of glutamine repeats (polyQ), making the mutant protein structurally unstable. This repeat confers one or more toxic functions to the mutant protein leading to neurodegeneration. Interestingly, for HD and SCA, the CAG repeat length underlies the clustering into adult-onset, early-onset and intermediate groups.
Despite the fact that the HD- and SCA-causing genes have been discovered for decades, there are currently no therapies to slow down, delay or prevent these diseases. In addition, it is unknown whether different therapies should be given to any of the three different disease clusters, or at what precise moment. There is also a lack of understanding on how to ethically guide genetic predictive decisions for these patients. These are important gaps that currently hamper the development of personalized treatments and ethical support for HD or SCA patients. An important contributing factor to this lack of knowledge is the scarcity of disease models for the three different clusters. These models are crucial to aid in the discovery of cell biological readouts but also to help predict individual patient outcomes, including response to treatment.
Therefore, CureQ intends to fill these important gaps by accomplishing the following aims:
- To collect biosamples from patients in all disease categories and generate accurate phenotypic of their clinical characteristics
- Develop patient-derived 2D and 3D culture models and define molecular disease landscapes in them that represent and model the disease trajectories in all disease categories.
- To find novel modulators of disease onset and progression in vitro.
- To provide ethical guidance for a responsible implementation of new prognostic information for carriers of neurodegenerative diseases and to formulate the conditions for implementation of prognostic information and personalized medicine in an ethically responsible way.
We will open research positions soon for PhD candidates. Please check our recruitment page for more information.